Morquio A Registry Study (MARS)
Research type
Research Study
Full title
Morquio A Registry Study (MARS)
IRAS ID
159401
Contact name
Saikat Santra
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Clinicaltrials.gov Identifier
Product reference, EMEA/H/C/0002779; Procedure number, EMEA/H/C/0002779/0000
Research summary
The Morquio A Registry Study (MARS) is an international observational program for patients with Morquio A disease. Morquio A disease is a rare inherited lysosomal storage disorder that causes difficulties with many different organ systems in the body – especially the musculoskeletal system, heart and lungs. The data collected by this registry are intended to allow a better understanding of the natural history of Morquio A disease by tracking the clinical outcomes of patients with Morquio A disease over time. All patients with a confirmed diagnosis of Morquio A disease may be eligible to participate in this registry.
A new intravenous enzyme replacement treatment (elosulfase alfa; Vimizim) has recently been licensed and as part of the conditions of the Global Marketing Authorisation, the MARS registry will also track the use of elosulfase alfa, specifically looking for treatment-related adverse events.
No experimental treatments or assessments are involved in this program. It is not required that patients receive elosulfase alfa to be eligible to participate in this program. Treating physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine follow-up.
The registry will have separate substudies specifically to monitor the effect of elosulfase alfa in patients who participated in the MOR 005 and MOR 007 clinical trials (including children under the age of 5 years) and in female patients who become pregnant (including assessing the outcome of any babies born to women receiving elosulfase alfa).
REC name
London - Central Research Ethics Committee
REC reference
14/LO/1263
Date of REC Opinion
11 Jul 2014
REC opinion
Favourable Opinion